Amsterdam Molecular Therapeutics Provides Business Update for the Third Quarter 2011

AMSTERDAM, November 17, 2011 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today provided its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2011.

3Q 2011 Highlights

Glybera® Marketing Authorisation Application filed for re-examination

Exclusive license signed with National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain indications

Dr. Carlos R. Camozzi appointed as Chief Medical Officer

CHMP Decision on Glybera and Re-organization

Glybera Marketing Authorisation Application re-examination:

Committee for Advanced Therapies (CAT), Scientific Advisory Group (SAG) and new Rapporteur/Co-Rapporteur all recommended Glybera approvable under exceptional circumstances

CHMP concluded that there are no significant safety issues with Glybera

CHMP concluded that insufficient evidence of clinical benefit means Glybera is not approvable at this time

AMT will focus development efforts and financial resources on three gene therapy programs: hemophilia B, GDNF and acute intermittent porphyria

Company remains committed to participating in the Sanfilippo B program, which is fully funded by a consortium led by Institut Pasteur

Further investment in and development of Glybera and Duchenne's muscular dystrophy programs suspended

AMT management is working in close consultation with the AMT Works Council to assess the impact of the review on the Company's workforce

Management and board believe that for AMT to remain a viable business it needs to reduce the headcount by 50% to 45 full-time employees

Business Update

The Company continues partnering discussions with pharma companies. At this time, AMT anticipates that it may be possible to conclude a partnership for its hemophilia B program during the first half of 2012, provided that additional funding can be secured in the meantime. Therefore, AMT is exploring all options with regard to the business and its financing, with support from existing shareholders. AMT is also looking at additional collaborations with academic groups, which would contribute towards the short-term funding of its GDNF program for up to three indications.

AMT's cash position* on September 30, 2011 amounted to € 3.8 million compared to € 9.1 million on June 30, 2011. The cash outflow in the third quarter of 2011, € 5.3 million compared to € 3.4 million in the prior year, mainly represented operational cash flow impacted by certain non-recurring items: outstanding debtors at September 30, 2011 included two amounts totaling € 0.7 million which were due to AMT in the third quarter. In addition, payments to two third parties under an old grant were triggered, giving rise to a cash outflow of € 0.75 million. Adjusting for these on a pro forma basis, AMT's cash position would have been € 5.25 million, in line with budget, and the third quarter cash outflow would have been € 3.85 million. AMT employed 83 permanent staff as of September 30, 2011. Total expenses in the third quarter of 2011 were € 3.7 million compared to € 4.7 million in the same period last year.

*The Company's cash position is composed of cash and cash equivalents.

Material events after September 30, 2011

Since September 30, 2011, AMT was informed of the outcome of the Glybera re-examination process and has consequently initiated a reorganization of the company, in each case as described above. On November 16, 2011, AMT announced that its hemophilia B program was granted Orphan designation in the European Union.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of several gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT to pursue its strategy of focusing on this sector of the industry. AMT was founded in 1998 and is based in Amsterdam. Further information can be found at http://www.amtbiopharma.com.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business. AMT expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law.